RecruitingNCT04872179
Blood
Alpha-Thalassemia
Also known as HbH disease, alpha-thal, hemoglobin H disease
Alpha-thalassemia results from deletions or mutations affecting one to four alpha-globin genes on chromosome 16, disrupting hemoglobin alpha-chain synthesis and causing a spectrum of disease from silent carrier state to hydrops fetalis. Hem
ORPHA:846 ↗Gene HBA1Gene HBA2Prevalence Carrier frequency up to 40% in endemic regions; HbH disease affects approximately 1 in 15,000 globallyOnset Birth to infancyAutosomal recessive
15
studies recruiting now
as of 7 Sept 2026
40
studies registered in total
as of 7 Sept 2026
12
countries with a recruiting site
as of 7 Sept 2026
8 Apr 2026
most recent study posted
among recruiting studies
Recruiting trials
RecruitingNCT03937817
Collection of Human Biospecimens for Basic and Clinical Research Into Globin Variants
RecruitingPhase 3NCT07517133
A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Dependent Thalassemia
RecruitingPhase 3NCT07506863
A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)
RecruitingPhase 2NCT05664737
A Study to Determine the Efficacy and Safety of Luspatercept in Adult Participants and to Evaluate the Safety and Pharmacokinetics in and Adolescent Participants With Alpha (α)-Thalassemia
Showing the 5 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.
See all 15 recruiting studiesWhere recruiting studies are running
Countries with at least one recruiting site among the studies above, 7 Sept 2026. Tap a country to search trials there.
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About Alpha-Thalassemia
Alpha-thalassemia results from deletions or mutations affecting one to four alpha-globin genes on chromosome 16, disrupting hemoglobin alpha-chain synthesis and causing a spectrum of disease from silent carrier state to hydrops fetalis. Hemoglobin H disease (three-gene deletion) causes moderate to severe hemolytic anemia, while four-gene deletion (Hb Bart hydrops fetalis) is typically fatal without in utero intervention. The condition is most prevalent in Southeast Asia, sub-Saharan Africa, and the Mediterranean basin.
Common clinical features
Hemolytic anemia ranging from mild to severe depending on gene deletion countSplenomegaly and hepatomegalyJaundice and neonatal hyperbilirubinemiaPallor and chronic fatigueHemoglobin H inclusion bodies visible on supravital stainingCholelithiasis from chronic hemolysisGrowth delay in severe formsHydrops fetalis in four-gene deletion (Hb Bart)
From Orphanet’s phenotype annotations (CC BY 4.0). Not a complete list.
Treatments being studied
1 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.
Phase 3Mitapivat
Before you apply
Things trial teams commonly ask about for Alpha-Thalassemia. Not eligibility rules; those are set by each study.
- Specify your genotype precisely (number of deleted alpha-globin genes and any non-deletion mutations) as trials distinguish between HbH disease, Hb Bart, and carrier states.
- Transfusion dependency status and splenectomy history are key eligibility criteria for emerging gene therapy trials targeting alpha-thalassemia.
- Newborn screening results and hemoglobin electrophoresis or HPLC records help confirm diagnosis for registry enrollment and trial screening.
Related conditions
Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).